[네이처] [사이언스] 제론사의 배아줄기세포 임상시험 포기에 관련된 기사

[죽순]

제론사가 인간을 대상으로한 배아줄기세포 임상시험을 포기한다고 발표한 것은 이 분야에 충격으로 받아들여졌습니다.

그러나 우리나라에서는 지나가는 단신 정도로만 취급을 했지요.

그동안 엄청난 돈을 쏟아부으면서 수정란 배아줄기세포를 수립하고 동물을 대상으로 한 실험을 마치고 FDA를 상대로 투쟁하여 인간을 대상으로한 임상시험을 허가받아 시행중이었는데, 갑자기 손을 떼겠다고 했으니 충격이 아닐 수 없지요.

앞장서서 길을 닦아 나가던 선구자가 배아줄기세포 사업을 접어버리겠다고 했으니 후발주자들에게는 더 많은 고난이 기다리고 있는 것일까요? 아니면 일등으로 앞장서 나갈 기회를 얻게 된 것일까요?

어쨌건 배아줄기세포를 이용한 난치병 치료라는 것의 효용성에 대하여 한번쯤은 다시 생각해 볼 기회를 가져야 할 것으로 봅니다. 배아줄기세포 연구에 대한 투자 열기가 식을까봐 쉬쉬하며 넘어갈 일은 아니지요.

[전에 했던 이야기]

미국 제론사 줄기세포 임상시험 포기

http://cafe.daum.net/46st/BRrk/101275

[ABC News] Geron Announcement Throws Stem Cell Research into Question - 줄기세포 시대의 상징적 종말을 알리는 신호?

http://cafe.daum.net/46st/BRrk/101284

지난주에 네이처와 사이언스에 이에 대한 글이 올라왔습니다.

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http://www.nature.com/news/stem-cell-pioneer-bows-out-1.9407

Stem-cell pioneer bows out

Geron halts first-of-its-kind clinical trial for spinal therapy.

22 November 2011

 

The first company to test a human embryonic stem-cell product in patients has become the first big player to bail out of the field. Last week’s move shook investor confidence and raised questions about whether the company had overreached itself, as well as underscoring just how difficult novel therapies are to develop.

Geron, based in Menlo Park, California, announced on 14 November that it would cease work on its stem-cell-therapy programmes to focus on its anti-cancer portfolio. “It’s kind of a heartbreak,” says Melissa Carpenter, principal of Carpenter Group Consulting in Seattle, Washington, who served as Geron’s director of stem-cell biology in the company’s early days. “It is truly unfortunate for the field that the first possible product didn’t get to go out of the gate.”

Human embryonic stem (ES) cells have the potential to turn into any of the body’s cell types, and so could replace defective tissue in myriad diseases. Geron chose to pursue a treatment for spinal-cord injury — an ambitious goal, not least because spinal damage involves many cell types. Some in the field speculate that Geron went forward with the programme, at least in part, because the neural-cell precursors it was testing are relatively easy to derive from human ES cells, and because dramatic results in animal studies impressed investors when the company needed funding. However, human trials are often the only way to test such unprecedented therapies.

Only four of a planned eight patients in Geron’s phase I trial have received injections of specialized cells derived from human ES cells. John Scarlett, who became Geron’s chief executive in September (see ‘Rough ride’), says that the company will continue to monitor enrolled patients, but will not recruit more. None of the four patients receiving stem-cell injections suffered serious adverse events, but there were also no hints that the therapy was working (although phase I trials are not designed to test for efficacy).

View Rough ride

REDUX/EYEVINE

The price of Geron’s stock fell by more than 30% at the news, from US$2.28 to $1.50 per share as Nature went to press. But the company estimates that discontinuing stem-cell research will save $25 million a year, allowing it to conduct half a dozen phase II clinical trials of its two cancer products in the next two years without raising additional funds. Cell-therapy studies would have taken longer and cost more.

Advanced Cell Technology (ACT) of Santa Monica, California, is now the only company conducting regulator-approved clinical trials involving human ES cells; these aim to treat degenerative eye diseases using specialized retinal cells.

Investors and patients are eager for reassurance that human ES cells have commercial and therapeutic potential, says Robert Lanza, chief scientific officer of ACT. “The field at this early point desperately needed a big success,” he says. “It certainly puts a lot of pressure on us to deliver.”

“Just because Geron is out, it doesn’t mean that other trials will slow down.”

Geron’s decision comes shortly after a ruling that products and processes involving human ES cells are not patentable in Europe (see Nature http://dx.doi.org/10.1038/news.2011.597; 2011), but analyst Reni Benjamin of investment bank Rodman & Renshaw in New York believes that Geron’s decision was unrelated. It was probably already in the works when long-term chief executive Thomas Okarma left the company abruptly in February, and reflects a change in business strategy rather than a verdict on cell therapies in general, he says. “Just because Geron is out, it doesn’t mean that other trials will slow down.”

Geron had invested heavily to bring human ES cells to clinical trials. It funded the studies leading to the cells’ derivation in 1998 (J. A. Thomson et al. Science 282, 1145–1147; 1998) and burned through cash while devising ways to manufacture specialized cells from the stem cells, as well as running extensive animal tests to show that the cells were safe enough to use in humans. These early efforts paved the way for others trying to move stem cells into the clinic. “It’s exponentially easier,” says Lanza. “We know exactly what [regulators] want.”

There may be other benefits for the field. Geron controls extensive intellectual property relevant to human ES-cell therapy, says Ken Taymor, a stem-cell patent expert at the University of California, Berkeley, and it may now be more willing to license this portfolio to help others pursuing such therapies.

Asked what other companies can learn from Geron’s decision, Michael West, who ran Geron from 1990 to 1998 and is currently chief executive of biotech company BioTime of Alameda, California, simply suggests: “Don’t be the first one out the door. The first one out the door gets all the arrows in his back.”

 

Journal name:

Nature

Volume:

479,

Pages:

459

Date published:

(24 November 2011)

DOI:

doi:10.1038/479459a

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http://www.sciencemag.org/content/334/6059/1043.summary

Embryonic Stem Cells

Researchers Mull Impact of Geron's Sudden Exit From Field

1. Jocelyn Kaiser*

Summary

The decision last week by the biotech company Geron to cancel the first-ever clinical trial using human embryonic stem cells (hESCs) and sell off its stem cell programs has shaken the biomedical research community. Some worry that Geron's announcement, which the company attributes to financial concerns, will dampen enthusiasm from other companies and the public for hESC research. But others see the half-completed trial as smoothing the way for future human studies.

사이언스에 올라온 글은 전문을 읽어보지는 못했습니다. 요약만 읽어보았어요.

p.s.

저 위 네이처 뉴스 번역문이 브릭에 올라왔네요.

http://bric.postech.ac.kr/trend/news/view_kisti.php?id=197876

[죽순]

NIH에 가장 많은 줄기세포주를 등록한 곳이 제론사인데 제론사가 엎어져버리는 바람에 배아줄기세포 임상시험을 하는 회사는 ACT 하나만 남았습니다.

제론사는 배아줄기세포 사업을 접었지만 ACT는 계속해 나갈 것이라고 합니다. 제론사가 임상시험을 중단한 이유는 재정적 이유라고 합니다. 그러나 작년 말 기준으로 제론사는 1800만 달러의 현금을 보유하고 있었습니다. 그리 자금 압박에 시달리는 정도는 아니었던 셈. 오히려 ACT사는 현금 보유고가 240만 달러인가 밖에 되지 않았어요.

제론사는 배아줄기세포 사업을 접음으로써 매년 250만 달러(300억원쯤)를 절약할 수 있게 되었다고 합니다.

[죽순]

수정합니다.

Despite relatively small numbers of cells required to test its hESCs in these trials and its ability to monitor early-on efficacy signs, ACT doesn’t have a lot of cash. The firm ended this year’s third quarter with cash and cash equivalents of $13.9 million, compared to $16.1 million as of June 30, 2011. By contrast, Geron completed the same period with over $180 million on hand.

올해 삼사분기 기준 현금 보유

제론사 1억8천만 달러
ACT 1천3백9십만달러

[Mr.kim]

제론사가 쩐이 더 많음에도, 그럼에도 불구하고..

[죽순]

매년 300억원씩 추가로 투자하는 경우 줄기세포 치료가 실용화되는 때까지 들어갈 돈과 성공 가능성 성공했을 때 벌어들이게 될 돈 등을 비교한 결과, 본전 생각은 나지만 지금이라도 접는 것이 남는 장사라는 판단이 섰기 때문에 줄기세포 사업을 접는 것이겠지요.

하여간에 난치병 환자들의 기대에는 찬물을 끼얹은 셈. 그래서 제론사를 비난하는 목소리도 많습니다. 제론사가 해 오던 임상시험은 척수마비 환자를 대상으로 한 것이었는데, 시험대상 선정 자체가 무모한 것이었다는 의견도 있습니다. 강원래나 수퍼맨의 크리스토퍼 리를 걷게 해 주겠다는 이야기는 먼 훗날이라면 모르겠지만 지금으로서는 황당한 이야기일 뿐이라는 것.

[죽순]

ACT에서 하고 있는 임상시험은 시각장애자를 위한 치료인데, 그것이라도 좋은 결과가 나오기를 바랍니다.

희망을 놓을 필요는 없겠지만 너무 큰 기대는 금물. 난치병 환자들에게 곧 뭐가 이루어질 것처럼 구라치는 행위는 올바른 행위가 아니라고 생각합니다.

[Mr.kim]

지금이라도 연구를 접는 것이 남는 장사.

[죽순]

제론사가 사기업이기 때문에 그런 결정을 했을 것입니다. 경영자의 판단이죠.

민간 투자는 위축되겠지만 미국에서도 세금을 들여 하는 연구는 지속될 것입니다.
세금으로 하는 연구라면 국민이나 시민들의 통제를 받아야 할 것입니다.
구라빨로 세금 축내는 사람들을 걸러내고 묵묵히 연구하는 연구자들을 지원하기 위해서는 민간에서도 감시의 눈을 번뜩여야 할 듯.

[죽순]

저 위 네이처 뉴스 번역문이 브릭에 올라왔네요.

http://bric.postech.ac.kr/trend/news/view_kisti.php?id=197876

[Mr.kim]

=3=3=3=3